A Study to Assess Efficacy, Safety, and Tolerability of P1101 in Adult Patients With PV

Polycythemia Vera Clinical Trial

Official title:

A Phase IIIb, Randomized, Open-Label, Parallel Group, Multicenter Study to Assess Efficacy, Safety, and Tolerability of Two Dosing Regimens of Ropeginterferon Alfa-2b-njft (P1101) in Adult Patients With Polycythemia Vera (PV)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05481151
• Other study ID #: ECLIPSE PV / A22-203
• Status: Recruiting
• Phase: Phase 3
• Start date: October 26, 2022
• Est. completion date: December 28, 2025

Study information

• Verified date: September 2023
• Source: PharmaEssentia
• Contact: Jewell Jessup, PhD
• Phone: 1-800-999-2449
• Email: clinicaltrials[@]pharmaessentia.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

A Study to Assess Efficacy, Safety, and Tolerability of P1101 in Adult Patients with PV

Description:

Polycythemia vera (PV) is the most common type of chronic myeloproliferative neoplasm (MPN), with an annual reported incidence of up to 2.6/100,000. This is a long-term debilitating and life-threatening disease because it is associated with the risk of thrombosis, bleeding, and progression to myelofibrosis (MF) and secondary acute myeloid leukemia (sAML) Ropeginterferon alfa-2b-njft (P1101), which gained US marketing authorization in November 2021, is the only interferon alfa approved for the treatment of PV. This study aims to evaluate the efficacy, tolerability, and safety of ropeginterferon alfa-2b-njft (P1101) in US and Canadian PV patients, utilizing an optimized dosing regimen.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 100
• Est. completion date: December 28, 2025
• Est. primary completion date: January 11, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

1. Male or female subjects aged =18 years at the time of signing the informed consent form

2. Subjects diagnosed with PV according to the 2008 or 2016 World Health Organization (WHO) criteria

3. Subjects with good liver function at screening, which is defined as total bilirubin =1.5 × upper limit of normal (ULN), international normalized ratio (INR) =1.5 × ULN, albumin >3.5 g/dL, alanine aminotransferase (ALT) =2.0 × ULN, and aspartate aminotransferase (AST) =2.0 × ULN

4. Hemoglobin (HGB) =10 g/dL for females, and HGB =11 g/dL for males at screening

5. Neutrophil count =1.5 × 10^9/L at screening

6. Creatinine clearance rate =40 mL/min at screening (according to the Cockcroft-Gault formula)

7. Males and females of childbearing potential, as well as all women <2 years after the onset of menopause, must agree to use an acceptable form of birth control until 60 days following the last dose of the study drug, and females must agree to not breastfeed during the study

8. Written informed consent obtained from the subject and ability for the subject to comply with the requirements of the study

Exclusion Criteria:

1. Any contraindications to interferon alfa or hypersensitivity to interferon alfa

2. Subjects who stopped prior to interferon alfa therapy due to low efficacy or poor tolerability

3. Subjects with severe or serious diseases that the Investigator determines may affect the subject's participation in this study

4. History of major organ transplantation

5. Pregnant or breastfeeding women

6. Subjects with any other diseases that the Investigator determines will affect the

study results or may weaken the compliance to protocol, including but not limited to:

1. Prior or current autoimmune thyroid disease (clinical symptoms of hyper- or hypo-thyroidism), except subjects with controlled thyroid replacement therapy, could be enrolled

2. Other documented autoimmune diseases (such as hepatitis, immune thrombocytopenia [ITP], scleroderma, psoriasis, or any autoimmune arthritis)

3. Clinically significant pulmonary infiltration, infectious pneumonia, and non-infectious pneumonia, or a past history of interstitial pneumonia at screening

4. Active infection with systemic manifestations (e.g., presence of bacteria, fungi, and/or human immunodeficiency virus [HIV] at screening, excluding hepatitis B [HBV] and/or hepatitis C [HCV] at screening)

5. Evidence of severe retinopathy (e.g., cytomegalovirus [CMV]-induced retinitis, macular degeneration) or clinically significant eye diseases (due to diabetes or hypertension)

6. History or presence of clinically relevant depression per Investigator's judgment

7. Previously had suicidal attempts or has any risk for suicidal tendency at screening

8. Poorly controlled diabetes defined as HbA1c >8.0% for at least 1 year

9. Active thromboembolic complications caused by PV and abdominal hemorrhage in the active phase

10. History of any malignancy within 5 years (except adequately treated non-melanoma skin cancer, prostate cancer status post resection with an undetectable prostate-specific antigen (PSA), curative treated in-situ cancer of the cervix, ductal carcinoma in situ (DCIS) of the breast, Stage 1 Grade 1 endometrial carcinoma, or other solid tumors including lymphomas (without bone marrow involvement) curatively treated with no evidence of disease for =2 years prior to study)

11. History of alcohol or drug abuse in the past year

12. History or evidence of post-polycythemia vera-myelofibrosis (PPV-MF), essential thrombocythemia, or any non-PV MPN

13. Presence of blast cells in the peripheral blood in the past 12 weeks

7. Use any investigational drug <4 weeks prior to the first dose of study drug, or not recovered from effects of prior administration of any investigational drug

8. Any subject requiring a legally authorized representative

Related Conditions & MeSH terms

• Polycythemia
• Polycythemia Vera

Intervention

Drug:
• P1101 (Ropeginterferon alfa-2b-njft)
Ropeginterferon alfa-2b-njft
• Ropeginterferon alfa-2b-njft (P1101)
Ropeginterferon alfa-2b-njft

Locations

Country	Name	City	State
Canada	Tom Baker Cancer Centre	Calgary	Alberta
Canada	Juravinski Cancer Center - Hamilton Health Sciences	Hamilton	Ontario
Canada	The Ottawa Hospital	Ottawa	Ontario
Canada	Princess Margaret Hospital	Toronto	Ontario
Canada	St. Michael's Hospital	Toronto	Ontario
Canada	St. Paul's Hospital	Vancouver	British Columbia
United States	American Oncology Partners of Maryland PA (Center for Cancer & Blood Disorders)	Bethesda	Maryland
United States	Montefiore Medical Center	Bronx	New York
United States	University of North Carolina Lineberger Comprehensive Cancer Center	Chapel Hill	North Carolina
United States	University of Virginia - Emily Couric Cancer Center	Charlottesville	Virginia
United States	Astera HealthCare	East Brunswick	New Jersey
United States	Fort Wayne Medical Oncology and Hematology	Fort Wayne	Indiana
United States	East Carolina University	Greenville	North Carolina
United States	Wake Forest Baptist Medical Center	High Point	North Carolina
United States	MD Anderson	Houston	Texas
United States	Baptist MD Anderson	Jacksonville	Florida
United States	University of Tennessee Health Science Center	Memphis	Tennessee
United States	Tulane University Medical Center	New Orleans	Louisiana
United States	Mount Sinai	New York	New York
United States	Mercy Health	Paducah	Kentucky
United States	Washington University School of Medicine	Saint Louis	Missouri
United States	University of Utah	Salt Lake City	Utah
United States	University of Kansas Medical Center	Westwood	Kansas

Sponsors (1)

Lead Sponsor	Collaborator
PharmaEssentia

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Compare efficacy, safety, and tolerability of P1101 utilizing 250-350-500 mcg compared to the current labeled dosing through assessing the proportion of subjects that are in a complete hematologic response at Week 24.	CHR is defined as hematocrit (HCT) <45%, white blood cell (WBC) count <10 × 10^9/L, platelets (PLT) =400 × 10^9/L in the absence of phlebotomy in the previous 12 weeks.	24 weeks